If approved, Eteplirsen would have been able to treat ~13% of the DMD population, based on specific mutations. Despite 12+ hours of hearings, including patient and advocate testimonials, the FDA found the trial to be too poorly designed with not enough concrete outcome evidence, thus the denial. The FDA went so far to state that “anecdote and emotions don’t change the data.”

While blame is easy to turf, and hindsight is 20/20, I think we (as advocates and health care professionals), have a lot we can learn from this to better prepare for the next proposal. I believe that there is a fair amount of short-sightedness in the clinical trial design (with understandable reasoning) in that many companies hope and expect a drug to work in 6-24 months (all companies included), while these diseases take a lifetime to affect boys. In this limited timeframe, mitigation of disease is subtle with current outcome measures. Overall, it would be more cost efficient to run a single successful large randomized control trial for a longer time, rather than multiple failed trials.

In all, it makes me appreciate what ImagingDMD does, using quantitative, longitudinal, and objective outcome measures like MRI, to assess the state of health.